Do we need authorized orphan drugs when compounded medications are available?

What is known and objective: Orphan drugs are used to diagnose, prevent or treat a rare disease. This Commentary aims to present a number of case studies questioning the need for designating compounded medications with a long history of effective use, which is well-supported by published clinical evidence.

Comment: Prior to the market introduction of orphan drugs, medication compounding was done in our hospital pharmacy for several rare diseases. Examples include amifampridine for the treatment of Lambert-Eaton myasthenic syndrome (Firdapse(®)), ibuprofen for the treatment of neonatal patent ductus arteriosus (Pedea(®)) and zinc acetate for the treatment of Wilson's disease (Wilzin(®)). Several 'non-orphan' pharmaceutical products, used off-label for the treatment of rare diseases, that became orphan medicinal products include Hydrea(®) for the treatment of sickle-cell syndrome (Siklos(®)) and Viagra(®) for the treatment of pulmonary arterial hypertension (Revatio(®)).

What is new and conclusion: In our opinion, as indicated by our examples, a better balance should be struck between the development of orphan drugs along the recently established regulatory pathways and the pragmatic use of pharmacy-compounded products and evidence-based off-label use of already available commercial products. Societal needs would be best met by focusing orphan drug development on rare diseases for which there is a high unmet medical need.